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C Fred LeMaistre

Publications and source records attributed to C Fred LeMaistre.

2 recordsLinked to original sources

What is quality in a transplant program?

Abstract Hematopoietic stem cell transplantation (HSCT) as a field of medicine has been subject to rapid development and evolution since its inception. Traditionally, HSCT has been used for therapy of a diverse group of malignancies, bone marrow failure states, and inherited disorders. The rapid evolution of transplantation technology coupled with the diverse outcomes associated with a heterogeneous group of patients has stymied the development of consensus over objective programmatic indicators of quality, especially as they pertain to outcomes. In some regard, the lack of consensus has caused transplant programs to respond to a more consumer-driven paradigm of evaluation. The community of providers of transplantation therapies has responded by establishing standards for accreditation of facilities and uniform presentation of programmatic data. Rapid acceptance of the need for meaningful quality programs to address all aspects of the transplant facility has moved HSCT to the forefront of implementing standards for medical practice. Because definition of optimal outcomes in HSCT is likely to remain elusive, it is imperative that providers involved with HSCT continue to take a leadership role in defining program quality through further research.

Hematopoietic Stem Cell Transplantation↗

High-dose immunosuppressive therapy and autologous peripheral blood stem cell transplantation for severe multiple sclerosis.

There were 26 patients enrolled in a pilot study of high-dose immunosuppressive therapy (HDIT) for severe multiple sclerosis (MS). Median baseline expanded disability status scale (EDSS) was 7.0 (range, 5.0-8.0). HDIT consisted of total body irradiation, cyclophosphamide, and antithymocyte globulin (ATG) and was followed by transplantation of autologous, granulocyte colony-stimulating factor (G-CSF)-mobilized CD34-selected stem cells. Regimen-related toxicities were mild. Because of bladder dysfunction, there were 8 infectious events of the lower urinary tract. One patient died from Epstein-Barr virus (EBV)-related posttransplantation lymphoproliferative disorder (PTLD) associated with a change from horse-derived to rabbit-derived ATG in the HDIT regimen. An engraftment syndrome characterized by noninfectious fever with or without rash developed in 13 of the first 18 patients and was associated in some cases with transient worsening of neurologic symptoms. There were 2 significant adverse neurologic events that occurred, including a flare of MS during mobilization and an episode of irreversible neurologic deterioration after HDIT associated with fever. With a median follow-up of 24 (range, 3-36) months, the Kaplan-Meier estimate of progression (>/= 1.0 point EDSS) at 3 years was 27%. Of 12 patients who had oligoclonal bands in the cerebrospinal fluid at baseline, 9 had persistence after HDIT. After HDIT, 4 patients developed new enhancing lesions on magnetic resonance imaging of the brain. The estimate of survival at 3 years was 91%. Important clinical issues in the use of HDIT and stem cell transplantation for MS were identified; however, modifications of the initial approaches appear to reduce treatment risks. This was a heterogeneous high-risk group, and a phase 3 study is planned to fully assess efficacy.

Adult↗